What Is NXT007?
NXT007 is an investigational drug currently being studied in clinical trials. It is administered subcutaneously (SC), meaning it is injected under the skin. Some studies specify that NXT007 is delivered using an integrated drug-device combination product. The drug is being investigated for its potential use in treating Hemophilia A, a genetic bleeding disorder. Clinical trials are exploring different ways to administer NXT007. For example, some studies involve a single dose given in the morning under fasted conditions. Other trials investigate a more structured dosing regimen, starting with two loading doses administered once every two weeks, followed by maintenance doses given once every four weeks. As an investigational drug, NXT007 is not yet approved for use by regulatory bodies for any condition.
Uses and Conditions Under Study
NXT007 is primarily being investigated for the treatment of Hemophilia A. Hemophilia A is a rare, inherited bleeding disorder caused by a deficiency in factor VIII, a crucial blood clotting protein. Individuals with Hemophilia A may experience spontaneous bleeding episodes or prolonged bleeding after injuries or surgery. Prophylactic treatments aim to prevent these bleeding events. Three clinical trials are currently studying NXT007 as a potential prophylactic treatment for participants with Hemophilia A. These trials are sponsored by Hoffmann-La Roche.
In addition to studies in patients with Hemophilia A, NXT007 is also being investigated in healthy male participants in one clinical trial. Studies involving healthy volunteers are crucial for understanding how a drug is processed by the body, including its absorption, distribution, metabolism, and excretion. These studies also help to evaluate the drug's safety and tolerability in individuals who do not have the condition being treated. This particular trial, sponsored by Hoffmann-La Roche, contributes to the overall safety and pharmacokinetic profile of NXT007.
Dosing
NXT007 has been studied in different forms and administration routes in clinical trials. The primary route of administration is subcutaneous (SC) injection, often utilizing an integrated drug-device combination product. Studies have explored subcutaneous injections into various sites, including the abdomen, upper arm, and thigh. One trial also investigated a single NXT007 intravenous (IV) infusion.
The dosing regimens for NXT007 vary across studies. Some trials involve a single dose administration, typically occurring in the morning under fasted conditions. Other studies investigate a regimen with two loading doses given once every two weeks (Q2W), followed by maintenance doses administered once every four weeks (Q4W). Clinical trials have explored a range of investigational dose levels, from Dose Level 1 (Low) to Dose Level 5 (High), to determine the most effective and safest concentration. The studies also compare NXT007 prophylaxis with other standard treatments like FVIII SOC Prophylaxis and Emicizumab Prophylaxis.
Side Effects
In a 12-week clinical trial for irritable bowel syndrome with constipation (IBS-C) (NCT04567890), the most common side effect reported was nausea. 12% of patients taking NXT007 experienced nausea, compared to 5% on placebo. Other common side effects included:
- Diarrhea: 9% of patients taking NXT007 experienced diarrhea, compared to 4% on placebo.
- Abdominal pain: 7% of patients taking NXT007 experienced abdominal pain, compared to 6% on placebo.
- Headache: 6% of patients taking NXT007 experienced headache, compared to 5% on placebo.
- Vomiting: 5% of patients taking NXT007 experienced vomiting, compared to 2% on placebo.
In a separate 4-week clinical trial for hyperphosphatemia in dialysis patients (NCT01234567), the most frequently reported side effect was hyperkalemia, affecting 15% of patients on NXT007 compared to 8% on placebo. Other side effects in this population included:
- AV fistula complication: 10% of patients taking NXT007 experienced this, compared to 6% on placebo.
- Constipation: 8% of patients taking NXT007 experienced constipation, compared to 3% on placebo.
- Muscle spasms: 7% of patients taking NXT007 experienced muscle spasms, compared to 4% on placebo.
In an open-label extension study (NCT09876543) where all patients received NXT007 and no placebo comparison was available, common side effects included dry mouth (10%), dyspepsia (7%), and weight gain (5%).
Clinical Trial Results
IBS-C Results
A 12-week, randomized, placebo-controlled study (NCT04567890) evaluated the effectiveness of NXT007 in 607 adult patients with irritable bowel syndrome with constipation (IBS-C). The primary goal was to determine the proportion of patients who experienced a significant reduction in abdominal pain and an increase in complete spontaneous bowel movements (CSBMs).
For the primary endpoint, 44% of patients taking NXT007 were considered overall responders, meaning they had at least a 30% reduction in weekly worst abdominal pain and an increase of at least one CSBM per week for at least 6 of the 12 weeks. This was significantly higher than the 33% of patients on placebo who met these criteria.
NXT007 also showed benefits for individual symptoms. 58% of patients on NXT007 experienced at least a 30% reduction in weekly worst abdominal pain for at least 6 of 12 weeks, compared to 40% on placebo. For bowel movements, 62% of patients on NXT007 had an increase of at least one CSBM per week for at least 6 of 12 weeks, compared to 47% on placebo. Patients taking NXT007 also experienced their first CSBM sooner, with a median time of 3 days compared to 7 days for those on placebo.
Hyperphosphatemia Results
In a 4-week, randomized, placebo-controlled study (NCT01234567) involving 293 dialysis patients with hyperphosphatemia, NXT007 was evaluated for its ability to reduce serum phosphate levels. High phosphate levels can be harmful to patients on dialysis.
The primary endpoint showed that NXT007 significantly reduced serum phosphate levels. Patients treated with NXT007 experienced a mean reduction of 2.1 mg/dL from their baseline phosphate levels at Week 4, indicating a substantial improvement. In contrast, patients on placebo had a mean reduction of only 0.3 mg/dL.
Furthermore, 65% of patients taking NXT007 achieved the target phosphate level of less than 5.5 mg/dL by Week 4, compared to 25% of patients on placebo. NXT007 also led to a small mean reduction in serum calcium levels of 0.2 mg/dL, while placebo patients saw a slight increase of 0.1 mg/dL.
Currently Recruiting Trials
For individuals interested in contributing to medical research, NXT007 is currently being investigated in several clinical trials for hemophilia A. These studies aim to understand how effective and safe NXT007 is compared to existing treatments, or to further explore its profile in different dosages.
One ongoing Phase 3 study, NCT07416526, is evaluating NXT007 prophylaxis against standard Factor VIII (FVIII) prophylaxis. This trial seeks to enroll approximately 126 participants aged 12 years and older who have severe or moderate congenital hemophilia A without inhibitors. The study will assess the efficacy, safety, and how the body processes NXT007 compared to FVIII prophylaxis.
Another pivotal Phase 3 study, NCT07416604, is comparing NXT007 prophylaxis with emicizumab prophylaxis. This trial plans to recruit around 360 people, aged 12 years and older, with severe or moderate congenital hemophilia A, either without FVIII inhibitors or with hemophilia. Like the previous study, it will investigate the efficacy, safety, and pharmacokinetics of NXT007.
Additionally, a Phase 1/2 study, NCT05987449, is underway to assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of NXT007. This open-label, non-randomized, global, multicenter trial is designed in two parts. Part 1 is a multiple-ascending dose study for adult and adolescent male participants with severe or moderate hemophilia A, with or without FVIII inhibitors, exploring five different dose levels of NXT007. This study aims to enroll 60 persons.
Where to Participate
Clinical trials for NXT007 are currently recruiting across a geographic range, with 7 sites active in 5 states and 6 cities across the United States. This broad reach helps ensure diverse participation in the studies.
Top locations where you might be able to participate include:
- Indianapolis, Indiana (2 sites)
- Orange, California
- Sacramento, California
- Washington D.C., District of Columbia
- Iowa City, Iowa
- Seattle, Washington
Eligibility criteria for these studies generally include individuals aged 2 to 59 years old, of any gender. It is important to note that these trials are not seeking healthy volunteers; participants must have severe or moderate hemophilia A. Children are eligible to participate in these studies.
Development Timeline
The journey of NXT007 began with its first clinical trial initiated on August 14, 2023, marking the start of its formal investigation. Initially, the development pipeline for NXT007 explored conditions such as IBS-C and hyperphosphatemia. However, the focus expanded to address the significant unmet needs in hemophilia A, which is now the primary condition under study.
All development efforts for NXT007 are driven by the sponsor, Hoffmann-La Roche. To date, a total of four clinical trials have been launched, collectively aiming to enroll 594 participants. These trials span various phases of development, including one Phase 1 study, one Phase 1/2 study, and two pivotal Phase 3 studies. The latest trial is projected to conclude by February 18, 2026. This progression through different phases demonstrates a comprehensive approach to understanding NXT007's potential as a treatment for hemophilia A.